Molecular Targets in Biliary Carcinogenesis and Implications for Therapy.

Biliary tract cancers (BTCs) encompass a group of invasive carcinomas, including cholangiocarcinoma (intrahepatic, perihilar, orextrahepatic), and gallbladder carcinoma. Approximately90% of patients present with advanced, unresectable disease and have a poor prognosis. The latest recommendation is to treat advanced ormetastatic disease with gemcitabine and cisplatin, although chemotherapy has recordedmodest survival benefits. Comprehension of the molecular basis of biliary carcinogenesis has resulted in experimental trials of targeted therapies in BTCs, with promising results. This review addresses the emerging role of targeted therapy in the treatment of BTCs. Findings from preclinical studies were reviewed and correlated with the outcomes of clinical trials that were undertaken to translate the laboratory discoveries.